A Phase 2b Study of Zagociguat in Patients With MELAS

Completed · Phase 2 · Has a placebo group

Conditions studied: Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)

In brief

PRIZM is a Phase 2b randomized, double-blind, placebo-controlled, 3-treatment, 2-period, crossover study evaluating the efficacy and safety of oral zagociguat 15 and 30 mg vs. placebo when administered daily for 12 weeks in participants with genetically and phenotypically defined MELAS.

Key facts

Study ID

NCT06402123

Run by

Tisento Therapeutics

People needed

43

Starts

2024-09-10

Expected to finish

2026-06-29

Last updated by the study team

2026-07-07

Who can join

Age: 18 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Signed consent form.
• 18 to 75 years of age.
• Diagnosed with MELAS based on the presence of each of the following criteria:
• A documented pathogenic variant in a mitochondrial DNA (mtDNA) gene.
• History of one or more stroke-like episodes (SLEs) with magnetic resonance imaging (MRI) findings consistent with stroke-like lesions.
• Scores below normal average on the iDSST and GMLT.
• Reports fatigue due to MELAS.
• Can complete at least 1 sit-to-stand in the 30-second test interval.
• Completes all at-home weekly activities independently during the Screening Period (caregiver may help set up device/app, log-in, etc.).
• Other criteria per the protocol.

You may not qualify if…

• Systolic blood pressure (BP) 90 mmHg or diastolic BP 60 mmHg.
• Orthostatic hypotension when measured after standing from a semi-recumbent/supine position.
• Active cancer significant enough to confound the results of this study.
• Severe gastrointestinal dysmotility that may impact participation.
• Recent history (within last 6 months) of platelet dysfunction, hemophilia, von Willebrand disease, coagulation disorder, other bleeding diathesis condition(s), or significant, nontraumatic bleeding episodes.
• History of spontaneous fracture(s) that in the investigator's opinion represents a safety risk for trial participation.
• Current use of prohibited medication (reviewed by investigator).
• Any medical or other condition that the investigator thinks would preclude study participation.
• Other exclusion criteria per protocol.

Where it is running

• UC San Diego - Altman Clinical and Translational Research Institute — La Jolla, California, United States
• Children's Hospital of Colorado — Aurora, Colorado, United States
• Rare Disease Research — Atlanta, Georgia, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Columbia University Irving Medical Center — New York, New York, United States
• Mount Sinai - Ichan School of Medicine — New York, New York, United States
• Akron Children's Hospital — Akron, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Baylor College of Medicine — Houston, Texas, United States
• University of Texas Medical School at Houston — Houston, Texas, United States
• Neuroscience Research Australia — Sydney, New South Wales, Australia
• Royal Melbourne Hospital — Melbourne, Victoria, Australia
• Shared Health/University of Manitoba — Winnipeg, Manitoba, Canada
• McMaster University Medical Center — Hamilton, Ontario, Canada
• University Hospital Bonn — Bonn, Germany
• Ludwig-Maximilians-University of Munich — Munich, Germany
• Neurologic Institute Carlo Besta of Milan — Milan, Italy
• Fondazione Policlinico Universitario Agostino Gemelli — Roma, Italy
• UCL Queen Square Institute of Neurology — London, United Kingdom
• Newcastle University — Newcastle upon Tyne, United Kingdom

Full record on ClinicalTrials.gov

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